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Once-weekly somapacitan in children with Noonan syndrome: randomized controlled phase 3 trial
Alexander A L Jorge1, Assunta Albanese2, Michael Højby3
1Genetic Endocrinology Unit (LIM25), Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (FMUSP), Sao Paulo 05403-010, Brazil.
Insights
Once-weekly somapacitan demonstrated superior height velocity compared to daily growth hormone (GH) injections in children with Noonan syndrome. This long-acting GH option showed a similar safety profile, offering a more convenient treatment for patients and caregivers.
Area of Science:
- Pediatric Endocrinology
- Genetics and Rare Diseases
- Pharmacology
Background:
- Noonan syndrome is a genetic disorder often treated with daily growth hormone (GH) injections, posing a significant treatment burden.
- Somapacitan is a novel, long-acting albumin-binding GH designed for once-weekly administration.
- Evaluating alternative GH therapies is crucial for improving treatment adherence and outcomes in pediatric patients.
Purpose of the Study:
- To assess the efficacy and safety of once-weekly somapacitan compared to daily GH in children with Noonan syndrome.
- To determine if somapacitan offers a non-inferior or superior treatment option for growth.
- To evaluate the tolerability and safety profile of somapacitan in this pediatric population.
Main Methods:
- A 52-week, randomized, open-label, active-controlled Phase 3 study (REAL8) involving 77 treatment-naïve children with Noonan syndrome.
- Participants were randomized 2:1 to receive either once-weekly somapacitan (0.24 mg/kg/week) or daily GH (0.050 mg/kg/day).
- Primary endpoint was annualized height velocity at week 52; secondary endpoints included changes in height standard deviation score and safety assessments.
Main Results:
- Somapacitan achieved a significantly higher mean annualized height velocity (10.4 cm/year) compared to daily GH (9.2 cm/year), confirming superiority (p<0.01).
- The change in height standard deviation score from baseline was greater with somapacitan (1.07) than with daily GH (0.75).
- Somapacitan was well-tolerated and exhibited a safety profile comparable to daily GH.
Conclusions:
- Once-weekly somapacitan is a safe and effective treatment for short stature in children with Noonan syndrome.
- Somapacitan demonstrated non-inferiority and superiority in height velocity compared to daily GH over 52 weeks.
- The similar safety and tolerability profiles suggest somapacitan as a viable, less burdensome alternative to daily GH therapy.
Objective:
Daily growth hormone (GH) injections are indicated for the treatment of short stature in children with Noonan syndrome, which presents a treatment burden for the child and their parents/caregivers. Somapacitan is a long-acting, reversible albumin-binding GH, developed for once-weekly administration. The objective of this study is to evaluate efficacy, safety, and tolerability of somapacitan versus daily GH in children living with Noonan syndrome.
Design:
REAL8 (NCT05330325) is a multinational, multicenter, randomized, open-labeled, active comparator, phase 3 basket study including four non-GH deficiency indications comprising a 52-week main phase and 104-week extension. Here, we present 52-week results from the REAL8 Noonan syndrome substudy.
Methods:
Seventy-seven GH-treatment-naïve, prepubertal boys (aged 2.5-11 years) and girls (aged 2.5-10 years) with Noonan syndrome were randomized 2:1 to somapacitan 0.24 mg/kg/week or daily GH 0.050 mg/kg/day, administered subcutaneously.
Results:
The primary endpoint, estimated mean annualized height velocity at week 52, was 10.4 cm/year for somapacitan versus 9.2 cm/year for daily GH (estimated treatment difference [ETD]: 1.2, 95% CI [0.32; 2.03]), confirming noninferiority and demonstrating superiority of somapacitan compared to daily GH (P < .01). The estimated change from baseline to week 52 in height standard deviation score was 1.07 and 0.75 for somapacitan and daily GH, respectively (ETD: 0.32, 95% CI [0.16; 0.48]). Somapacitan was well tolerated and had a similar safety profile to daily GH.
Conclusions:
Once-weekly somapacitan was confirmed as noninferior and demonstrated superiority to daily GH in HV after 52 weeks of treatment in treatment-naïve children living with Noonan syndrome. Similar safety profiles and tolerability were observed for both groups.
Clinical Trial Registration:
NCT05330325.
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